3 results
Approved WMOCompleted
The study will be performed in 3 parts, Parts 1, 2 and 3. In all parts, the purpose is to investigate to what extent APX001 is tolerated. In addition, it will be investigated how quickly and to what extent APX001 is absorbed and eliminated from the…
Approved WMORecruiting
Primary Objective: Assess the ability of MRCP+ and LMS to detect change in total biliary volume and cT1 value, 8 weeks after endoscopic treatment of dominant strictures, by a clinical decision rule.Secondary Objective(s): Comparison of baseline MRCP…
Approved WMORecruiting
The primary objective of this study is to evaluate the efficacy of olipudase alfa in adult patients with acid sphingomyelinase deficiency.